Costs and benefits of early access to new cancer drugs through the U.S. Food and Drug Administration accelerated approval pathway

Naci, Huseyin ORCID: 0000-0002-7192-5751, Shahzad, Mahnum, Murphy, Peter, Zhang, Yichen, Costa, Rebecca, Ross, Joseph S. and Wagner, Anita K. (2025) Costs and benefits of early access to new cancer drugs through the U.S. Food and Drug Administration accelerated approval pathway. BMJ Medicine. ISSN 2754-0413 (In Press)

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Abstract

Objective: To evaluate the survival gains and additional Medicare spending associated with early access to new cancer drugs granted accelerated approval by the U.S. Food and Drug Administration (FDA), compared with access following completion of confirmatory trials. Design: Retrospective observational study and economic evaluation. Setting: U.S. Medicare program, which provides health insurance for adults aged 65 years and older. Participants: Medicare beneficiaries who received new cancer drug indications from the time of FDA accelerated approval to conversion to regular approval, withdrawal, or 31 December 2020. Exposure: Use of cancer drugs for indications that initially received FDA accelerated approval between 1 January 2012 and 31 December 2020. Main outcomes and measures: The primary outcomes were life-year gains and additional Medicare spending associated with cancer drug indications that initially received FDA accelerated approval. We first determined whether each indication demonstrated an overall survival benefit. We then developed a partitioned survival model to estimate the life-year gains and incremental Medicare spending attributable to early access. Beneficiaries who received accelerated approval indications were followed for survival and spending outcomes until 31 December 2022. Results: An estimated 178,708 Medicare beneficiaries received 90 new cancer drugs from the time of accelerated approval to conversion to regular approval, withdrawal, or 31 December 2020. 30,374 (17.0%) beneficiaries received drugs for indications that were ultimately withdrawn, 5,574 (3.1%) for indications that remained under accelerated approval, and 142,760 (79.9%) for indications that were subsequently converted to regular approval. Overall, 80,885 (45.3%) beneficiaries received drugs for indications that demonstrated an overall survival benefit. Between 2012 and 2022, use of these drug indications was associated with an estimated 76,164 life-years gained. Only 3 accelerated approvals (nivolumab for melanoma, pembrolizumab for non-small cell lung cancer, and pemetrexed for non-small cell-lung cancer) accounted for 52,107 (68.4%) of the total life-year gains. The estimated additional cost of early access to Medicare was $20.1 billion, corresponding to a mean spending of $263,371 (95% CI: $180,139 to $373,884) per life-year gained. Additional Medicare spending per life-year gained ranged from $25,947 (95% CI: $18,174 to $39,829) for melanoma indications to $4.5 million (95% CI: $3.1 to $12.2 million) for breast cancer indications. Conclusions: This study examines the trade-off between the benefits of earlier access to cancer drugs through the FDA’s accelerated approval pathway and the uncertainty surrounding their clinical efficacy at the time of market entry. Accelerated approval yielded uneven survival returns, with a small number of drugs accounting for the majority of life-year gains. Medicare incurred substantial costs for treatments that, in over half of cases, did not provide an overall survival benefit.

Item Type:	Article
Additional Information:	© 2025 The Author(s)
Divisions:	Health Policy
Date Deposited:	17 Sep 2025 11:00
Last Modified:	17 Sep 2025 11:06
URI:	http://eprints.lse.ac.uk/id/eprint/129544

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